Getting a new medication to patients: Not a piece of cake - Prof Ingrid Scheffer скачать в хорошем качестве

Getting a new medication to patients: Not a piece of cake - Prof Ingrid Scheffer

In this compelling presentation, Professor Ingrid Scheffer takes us behind the scenes of what it really takes to bring a new epilepsy drug—from early trials to public access—for patients with Dravet syndrome and developmental and epileptic encephalopathies (DEEs). With deep personal and clinical insight, she shares the 10+ year journey of bringing fenfluramine to Australia and introduces a promising new therapy. Prof. Scheffer discusses how rigorous clinical trials, health economics, advocacy, and persistence shaped drug availability for patients who had previously exhausted all options. Through patient stories, data, and real-world outcomes—including the transformative impact on children like Gemma—this talk illustrates both the scientific and human sides of therapeutic innovation. 💊 Highlights include: The re-emergence of fenfluramine as an effective DEE therapy Challenges with pharmaceutical approval and access in Australia Trial design: placebo, blinding, and why evidence matters Health economic analysis and hospital cost savings Australia’s leadership in global clinical trials A must-watch for clinicians, researchers, patient advocates, and families—this video underscores why breakthroughs take more than science—they take community, courage, and tenacity.